FDA Should Exercise Regulatory Flexibility in Review of Elamipretide NDA Submission

On September 14, 2021, we released Abe’s Story featuring one of our #BarthStrong families. The video features Abe’s parents, Bryan and Erin, as well as footage of Abe, providing a glimpse of what it is like to live with Barth syndrome and the hope the family holds for treatment options.

ABE'S STORY

In addition, we published in STAT News about the vital importance of the FDA’s flexibility and openness to incorporating patients’ perspectives in ultra-rare disease review processes. In our publication, we ask the FDA to accept and exercise appropriate regulatory flexibility in their review of the elamipretide NDA submission.

We need your help!

THE FDA LISTENS TO SOCIAL MEDIA AND WE NEED THEM TO HEAR US! WE ARE ASKING OUR BSF FAMILIES AND FRIENDS TO DO THE FOLLOWING:

1. SHARE BSF’S SOCIAL MEDIA POSTS HIGHLIGHTING ABE’S STORY AND THE STAT NEWS PUBLICATION (Facebook, Instagram, Twitter)
2. LIKE ABE’S VIDEO on YouTube
3. CALL UPON YOUR FAMILY MEMBERS, NEIGHBORS, FRIENDS AND COLLEAGUES TO SHARE THESE LINKS AS WELL

If each of us in our global community takes these steps, we can make a big statement that we hope the FDA will hear!!

If you are not online and still want your voice heard, submit a letter to Shelley Bowen at shelley.bowen@barthsyndrome.org by Monday, September 20th.

Why is your help so urgent?

Stealth BioTherapeutics’ announced their submission to the FDA of their new drug application (NDA) for elamipretide in Barth syndrome on Tuesday, Aug. 24, 2021. The FDA has 60 days to decide to accept the submission, yet there is still a great deal of uncertainty about the outcome. If FDA does not file the application, people living with Barth syndrome could be denied the opportunity to access elamipretide. 

Together, the BSF community has done so much to advocate for access to treatments, including:

• Holding our Patient Focused Drug Development (PFDD) Meeting
• Mobilizing individuals and families to testify before FDA several times
• Organizing a listening session with FDA where they heard from Barth patients and caregivers about our tolerance for the uncertainty of benefit (meaning that a drug might not work equally well, or even at all, for some with Barth syndrome)
• Signing and asking others to support a global petition, gathering over 4,200 signatures requesting that Stealth submit an NDA (which they now have done) and that FDA file and review it (that is what we are still working on)
• Asking clinicians who really understand Barth syndrome to sign a letter to FDA supporting the NDA submission, filing and review
• Actively participating in many sponsor meetings with FDA by submitting oral and written statements
• Directly writing the FDA numerous times on behalf of all of those affected by Barth syndrome with additional details about our patients’ lives, concerns, needs and hopes

Currently, there are ZERO FDA-approved therapies for use in Barth syndrome. This is our chance to change this statistic!! There is power in numbers, and we are #BarthStrong when we work together.